F. Hoffman-La Roche, PTC and the SMA Foundation recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA): “In November 2014, Roche initiated the Moonfish trial (Phase 1b/2a) based on supportive results from a trial in healthy […]
Update on Clinical Development of RG7800 Read More »
Over the past few months, Cure SMA has announced a total of $1.85 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for: Basic research,
Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015 Read More »
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital. Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at Nationwide Children’s, and his team have made promising strides toward therapies
The US House of Representatives Energy and Commerce Committee has released a new 21st Century Cures discussion draft. This is a revised and expanded version of the first draft that was released in January. The 21st Century Cures initiative was created to investigate how the government can help “accelerate the discovery, development, and delivery of
New 21st Century Cures Discussion Draft Released Read More »
